Universal base editing for hemophilia B
Nemekhbayar Baatartsogt, Yuji Kashiwakura, Takafumi Hiramoto, Rina Ito, Rikako Sato, Yasumitsu Nagao, Hina Naruoka, Haruka Takata, Morisada Hayakawa, Khishigjargal Batjargal, Tomoki Togashi, Atsushi Hoshino, Taro Shimizu, Yusuke Sato, Tatsuhiro Ishida, , Tsukasa Ohmori
This study uses base editing to create a universal FIX variant for hemophilia B treatment, increasing FIX activity in cell and mouse models.
Blood · Oct 23, 2025Peer-reviewedBioengineering